By: Margaret Hamburg, M.D.
There’s an old maxim that you get what you pay for. That’s certainly been the case with FDA’s highly successful drug user fee program. Under this program, industry agreed to pay fees to help fund a portion of FDA’s drug review activities and in exchange, FDA agreed to meet specific performance metrics like reviewing a specific percentage of product submissions within a certain time period. The program was launched in the early 1990s at a time when FDA lacked sufficient staff to perform timely reviews or develop procedures to make the process more rigorous, consistent and predictable. As a result, access to new medicines for U.S. patients lagged behind other countries. User fees meant FDA could hire additional reviewers and support staff even when budgets were tight. The upshot: the agency was able to conduct reviews in a timely fashion, revolutionizing the drug approval process.
Since it became law 20 years ago, the Prescription Drug User Fee Act or PDUFA has produced significant benefits for public health, reversing the drug lag and providing patients faster access to over 1,500 new drugs. As a result of the continued investment of PDUFA resources, the United States now leads the world in first introduction of novel drugs.
PDUFA must be reauthorized every five years and the latest program, known as PDUFA IV expires on Sept. 30. Working collaboratively with industry, patient groups and consumer groups, FDA came up with a set of recommendations for a PDUFA V that Health and Human Services Secretary Kathleen Sebelius is sending to Congress today.
Under these recommendations, fees paid by industry would support continued timely review of critical prescription drugs plus some important enhancements for patients and industry including a commitment to advance the development of drugs for rare diseases, provide for enhanced communication with small companies, and adoption of some cutting-edge approaches to the use of data that will improve drug development times.
PDUFA has been so successful that FDA followed a similar model in developing recommendations for two new user fee programs – one is for generic drugs, copies of brand-name drugs and the other for products that are shown to be “biosimilar to” or “interchangeable with” an FDA-licensed biological product – such as a protein, a vaccine or a blood component.
Both programs would offer a net win for consumers and patients, offering more choice and lower-cost products.
To my way of thinking, these significant proposals headed to Congress today demonstrate what can be achieved when industry, patient groups and FDA put their heads together and work towards a common goal.
Margaret Hamburg, M.D., is Commissioner of the U. S. Food and Drug Administration.